Treatment with the antiplatelet agent prasugrel does not significantly reduce the rate of pain crises or severe lung complications in children with sickle cell disease, according to a report published in the New England Journal of Medicine describing one of the largest and most geographically diverse international clinical trials on sickle cell disease to date.

Preclinical data unveiled across four studies presented at the 57th annual meeting of the American Society of Hematology highlight four potential treatment opportunities for acute myeloid leukemia (AML), a blood cancer accounting for approximately 20 percent of all childhood leukemias and 32 percent of adult leukemias.

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology.

Prophylactic antibiotics significantly reduce the risk of serious bacterial infections in children during the critical first month of treatment for acute lymphoblastic leukemia (ALL), the most common childhood cancer, according to a U.S. and Canadian study led by investigators from Dana-Farber/Boston Children's. While the overall cure rate for ALL is high, about one to two percent of children with this diagnosis die during the first month of therapy from treatment complications, primarily infection-related.

Turning off a single gene leads to a roughly three-to-five-fold gain in the yield of laboratory methods for producing red blood cells from stem cells. These findings, published in Cell Stem Cell, suggest a way to cost-effectively manufacture red blood cells from stem cells; the patients who could potentially benefit include those who cannot use blood currently available in blood banks.

Disparities in outcomes for children with retinoblastoma — a rare eye tumor usually discovered in routine pediatric check-ups — suggest unequal access to primary care, researchers from Dana-Farber/Boston Children’s report in a study published in the journal JAMA Pediatrics. Although virtually all the children in the study survived, Hispanic children and children who lived in disadvantaged areas were more likely to lose an eye due to late diagnosis.

Almost one-third of families whose children were being treated for cancer faced food, housing or energy insecurity and one-quarter lost more than 40 percent of household income, according to a new study from Dana-Farber/Boston Children’s. The study, which was published in the journal Pediatric Blood & Cancer, follows emerging research in pediatric oncology finding that low-income status predicts poor adherence to oral chemotherapy and decreased overall survival.

Researchers from Dana-Farber/Boston Children's, using CRISPR-based gene editing tools, have found that changes to a small stretch of DNA may circumvent the genetic defect behind sickle cell disease (SCD). The discovery, published in the journal Nature, creates a path for developing gene editing approaches for treating SCD and other hemoglobin disorders, such as thalassemia.

More than two-thirds of adolescents and young adults dying of cancer utilized one or more aggressive interventions in the last month of life, according to a retrospective study from researchers at Dana-Farber/Boston Children’s and Kaiser Permanente Southern California.